小柯机器人

美国波士顿儿童医院Adrienne G. Randolph团队研究了儿童多系统炎症综合征的初步治疗方案和效果。这一研究成果于2021年6月16日发表在《新英格兰医学杂志》上。

为了评价免疫调节药物治疗儿童多系统炎症综合征（MIS-C）的实际疗效并指导治疗，2020年3月15日至10月31日，研究组对美国58家医院收治的年龄小于21岁的MIS-C住院患者的监测数据进行了分析。

采用倾向评分匹配法和逆概率加权法评估静脉注射免疫球蛋白（IVIG）加糖皮质激素与单纯IVIG初始免疫调节治疗的有效性，并对MIS-C基线严重程度和人口统计学特征进行校正。主要结局是第2天或之后的心血管功能障碍（左心室功能障碍或使用血管升压药导致休克的综合结局）。次要结局包括主要结局的组成部分，第一天或之后接受辅助治疗，第二天或之后持续或复发发热。

共有518例MIS-C患者（中位年龄8.7岁）接受至少一种免疫调节治疗，75%的患者此前健康，其中9人死亡。在倾向评分匹配分析中，首次接受IVIG加糖皮质激素治疗（103例）在第2天或之后心血管功能障碍的风险为17%，显著低于单纯接受IVIG治疗（103例，31%）。

接受IVIG加糖皮质激素治疗的患者与单纯IVIG相比，综合结局各组分的风险也较低：分别有8%和17%的患者出现左心室功能障碍，13%和24%的患者使用血管加压药后导致休克。接受IVIG加糖皮质激素治疗的患者有34%使用辅助治疗，显著低于单纯接受IVIG的患者（70%）；但两组的发热风险相差不大，分别为31%和40%。逆概率加权分析证实了倾向得分匹配分析的结果。

研究结果表明，对于患有MIS-C的儿童和青少年，初次使用IVIG加糖皮质激素治疗与单独使用IVIG相比，新发或持续性心血管功能障碍的风险显著降低。

附：英文原文

Title: Multisystem Inflammatory Syndrome in Children — Initial Therapy and Outcomes

Author: Mary Beth F. Son, M.D.,, Nancy Murray, M.Sc.,, Kevin Friedman, M.D.,, Cameron C. Young,, Margaret M. Newhams, M.P.H.,, Leora R. Feldstein, Ph.D.,, Laura L. Loftis, M.D.,, Keiko M. Tarquinio, M.D.,, Aalok R. Singh, M.D.,, Sabrina M. Heidemann, M.D.,, Vijaya L. Soma, M.D.,, Becky J. Riggs, M.D.,, Julie C. Fitzgerald, M.D.,, Michele Kong, M.D.,, Sule Doymaz, M.D.,, John S. Giuliano, Jr., M.D.,, Michael A. Keenaghan, M.D.,, Janet R. Hume, M.D.,, Charlotte V. Hobbs, M.D.,, Jennifer E. Schuster, M.D.,, Katharine N. Clouser, M.D.,, Mark W. Hall, M.D.,, Lincoln S. Smith, M.D.,, Steven M. Horwitz, M.D.,, Stephanie P. Schwartz, M.D.,, Katherine Irby, M.D.,, Tamara T. Bradford, M.D.,, Aline B. Maddux, M.D.,, Christopher J. Babbitt, M.D.,, Courtney M. Rowan, M.D.,, Gwenn E. McLaughlin, M.D.,, Phoebe H. Yager, M.D.,, Mia Maamari, M.D.,, Elizabeth H. Mack, M.D.,, Christopher L. Carroll, M.D.,, Vicki L. Montgomery, M.D.,, Natasha B. Halasa, M.D.,, Natalie Z. Cvijanovich, M.D.,, Bria M. Coates, M.D.,, Charles E. Rose, Ph.D.,, Jane W. Newburger, M.D., M.P.H.,, Manish M. Patel, M.D.,, and Adrienne G. Randolph, M.D.

Issue&Volume: 2021-06-16

Abstract:

BACKGROUND

The assessment of real-world effectiveness of immunomodulatory medications for multisystem inflammatory syndrome in children (MIS-C) may guide therapy.

METHODS

We analyzed surveillance data on inpatients younger than 21 years of age who had MIS-C and were admitted to 1 of 58 U.S. hospitals between March 15 and October 31, 2020. The effectiveness of initial immunomodulatory therapy (day 0, indicating the first day any such therapy for MIS-C was given) with intravenous immune globulin (IVIG) plus glucocorticoids, as compared with IVIG alone, was evaluated with propensity-score matching and inverse probability weighting, with adjustment for baseline MIS-C severity and demographic characteristics. The primary outcome was cardiovascular dysfunction (a composite of left ventricular dysfunction or shock resulting in the use of vasopressors) on or after day 2. Secondary outcomes included the components of the primary outcome, the receipt of adjunctive treatment (glucocorticoids in patients not already receiving glucocorticoids on day 0, a biologic, or a second dose of IVIG) on or after day 1, and persistent or recurrent fever on or after day 2.

RESULTS

A total of 518 patients with MIS-C (median age, 8.7 years) received at least one immunomodulatory therapy; 75% had been previously healthy, and 9 died. In the propensity-score–matched analysis, initial treatment with IVIG plus glucocorticoids (103 patients) was associated with a lower risk of cardiovascular dysfunction on or after day 2 than IVIG alone (103 patients) (17% vs. 31%; risk ratio, 0.56; 95% confidence interval [CI], 0.34 to 0.94). The risks of the components of the composite outcome were also lower among those who received IVIG plus glucocorticoids: left ventricular dysfunction occurred in 8% and 17% of the patients, respectively (risk ratio, 0.46; 95% CI, 0.19 to 1.15), and shock resulting in vasopressor use in 13% and 24% (risk ratio, 0.54; 95% CI, 0.29 to 1.00). The use of adjunctive therapy was lower among patients who received IVIG plus glucocorticoids than among those who received IVIG alone (34% vs. 70%; risk ratio, 0.49; 95% CI, 0.36 to 0.65), but the risk of fever was unaffected (31% and 40%, respectively; risk ratio, 0.78; 95% CI, 0.53 to 1.13). The inverse-probability-weighted analysis confirmed the results of the propensity-score–matched analysis.

CONCLUSIONS

Among children and adolescents with MIS-C, initial treatment with IVIG plus glucocorticoids was associated with a lower risk of new or persistent cardiovascular dysfunction than IVIG alone.

DOI: 10.1056/NEJMoa2102605

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa2102605

The New England Journal of Medicine：《新英格兰医学杂志》，创刊于1812年。隶属于美国麻省医学协会，最新IF：176.079
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